-MeiraGTx Receives
-
-MeiraGTx Remains Eligible for
LONDON and NEW YORK,
“XLRP is one of the most common and severe forms of retinitis pigmentosa, with no approved treatment options, and with most patients completely losing sight by the fourth decade of their lives,” said
About Botaretigene Sparoparvovec
Botaretigene sparoparvovec, formerly referred to as AAV-RPGR, is an investigational gene therapy for the treatment of patients with XLRP caused by disease-causing variants in the eye-specific form of the RPGR gene (RPGR ORF15). Botaretigene sparoparvovec is designed to deliver functional copies of the RPGR gene to the retina in order to improve and preserve vision. The Lumeos trial (NCT04671433) is a Phase 3 randomized, controlled study of botaretigene sparoparvovec for the treatment of XLRP associated with variants in the RPGR gene. Botaretigene sparoparvovec has been granted Fast Track and Orphan Drug designations by the FDA and PRIority MEdicines (PRIME), Advanced Therapy Medicinal Product (ATMP) and Orphan designations by the
About X-Linked Retinitis Pigmentosa (XLRP)
XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.
About MeiraGTx
For more information, please visit www.meiragtx.com.
Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of botaretigene sparoparvovec, the Phase 3 Lumeos clinical trial of botaretigene sparoparvovec and the achievement of milestones or regulatory approvals, including in light of the COVID-19 pandemic, as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended
Contacts
Investors:
Investors@meiragtx.com
Media:
jbraco@lifescicomms.com
Source:
2022 GlobeNewswire, Inc., source